On September 17, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported that the European Commission (EC) has approved ROMVIMZA (vimseltinib) in the European Union (EU) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability (Press release, Deciphera Pharmaceuticals, SEP 17, 2025, View Source [SID1234656040]).

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"The European Commission’s approval of vimseltinib for TGCT is a significant milestone for Deciphera, ONO, and TGCT patients across the European Union who are in need of a non-invasive treatment option. We are excited to leverage our global commercial infrastructure to bring vimseltinib to these patients," said Ryota Udagawa, President and Chief Executive Officer of Deciphera. "We look forward to working with health authorities to ensure all eligible patients who can benefit from vimseltinib have access as quickly as possible."

"This is welcome news for the TGCT community as vimseltinib is now the first approved therapy for TGCT in Europe," said Jean-Yves Blay, M.D., Ph.D., Leon Berard Center. "TGCT can significantly impact the daily lives of patients by causing pain, stiffness and mobility limitations. Vimseltinib is a differentiated treatment that has demonstrated the ability to address these unmet patient needs while remaining well-tolerated."

The EC approval is supported by compelling efficacy and safety results from the pivotal Phase 3 MOTION study of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of vimseltinib1. The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 251. The secondary endpoint was supported by statistically significant and clinically meaningful improvements versus placebo in all six key secondary endpoints assessed at Week 25 including objective response rate (ORR) by tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain1. In a descriptive analysis at Week 97, 23% (n=19/83) of the patients randomized to receive vimseltinib had best overall response of complete response (CR) according to RECIST v1.1, as assessed by blind independent radiological review (IRR), with a median time to CR of 11.5 months1. The safety profile of vimseltinib is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial1. For a full list of side effects and information on dosage and administration and other precautions, please refer to the Summary of Product Characteristics for further information.

About Tenosynovial Giant Cell Tumor (TGCT)

TGCT is caused by a translocation in colony-stimulating factor 1 (CSF1) gene resulting in overexpression of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion.2 TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS). TGCT is a rare, locally aggressive neoplasm that can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity, systemic treatment options are limited and a new therapeutic option for TGCT is needed.

On September 17, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported that the updated long-term follow-up results of Phase I clinical trial of zevorcabtagene autoleucel (zevor-cel, R&D code: CT053, an autologous CAR T-cell product targeting BCMA) have been presented as a poster at the 22nd International Myeloma Society ("IMS") Annual Meeting (Press release, Carsgen Therapeutics, SEP 17, 2025, View Source [SID1234656039]). The poster was titled "Long term Follow-up of Zevor-cel in Patients with Relapsed/Refractory Multiple Myeloma" (Abstract number: PA-029).

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In this study, a total of 14 patients with relapsed or refractory multiple myeloma (R/R MM) received a single infusion of zevor-cel. As of February 22, 2025, the median follow-up duration was 53.3 months (range:14.8, 63.5).

Regarding safety: There were no reports of ≥Grade 3 cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), delayed neurotoxicities, second primary malignancy or other delayed AEs on the study.

Regarding efficacy: The overall response rate was 100% (95% CI: 76.8, 100.0) with 11 (78.6%) patients achieving complete response (CR) or stringent complete response (sCR). All patients who achieved CR or better were minimal residual disease (MRD) negative at 10−5 threshold. One patient remained in sCR at 59.3 months in the study. The median progression-free survival (mPFS) and the median duration of response (mDoR) were 44.1 months and 43.2 months in CR/sCR patients, respectively. The median overall survival (OS) was not reached. The proportion of patients surviving at 24, 36, 48 and 60 months after infusion were 100%, 92.3%, 84.6% and 76.9%, respectively.

At approximately 5 years of follow-up, zevor-cel demonstrates manageable safety profile while eliciting deep and durable responses in R/R MM patients.

About Zevor-cel

Zevor-cel is a fully human, autologous BCMA CAR T-cell product for the treatment of Multiple Myeloma (MM). Zevor-cel was approved by the NMPA on February 23, 2024 for the treatment of adult patients with R/R MM who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019.

On September 17, 2025 Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, reported further encouraging efficacy data from its Phase 1b clinical trial evaluating azer-cel (azercabtagene zapreleucel) in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), an aggressive form of blood cancer (Press release, Imugene, SEP 17, 2025, https://mcusercontent.com/e38c43331936a9627acb6427c/files/eb0aad51-27f2-1fa7-4e15-300441221a2f/Overall_Response_Rate_increases_to_81_in_azer_cel_trial.pdf [SID1234656038]).

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In August 2025, Imugene announced that a total of eleven out of fourteen patients had achieved an ORR of 79%, defined as either Complete Response, (the disappearance of signs of cancer in response to treatment) or Partial Response, (defined as cancer reduction by at least 50%). Since then, two new patients have become evaluable for responses with both achieving a Partial Response and another patient transitioning from PR to CR at Day 90 scan evaluation increasing the best ORR to 81% with thirteen out of sixteen patients showing response to treatment. The Complete Response (CR) rate continues to evolve as enrollment progresses and patients transition from partial to complete response, with an average time to best response seen in 1–3 months. The durability of response is also deepening in patients treated with azer-cel in combination with interleukin-2 (IL-2).

Azer-cel is being developed as a potential allogeneic, off-the-shelf, CAR T-cell therapy, addressing key limitations of approved autologous CAR T drugs, including geographical access to treatment centres, manufacturing complexity and time to receive treatment (on-demand).

Imugene is actively enrolling patients to the Phase 1b azer-cel trial at ten US sites with up to six sites in Australia planned, after the first Australian patient was dosed in January 2025 at Royal Prince Alfred Hospital in Sydney, resulting in a Complete Response.

About the Phase 1b azer-cel trial

The azer-cel allogeneic CAR T trial is an ongoing, open-label, multi-centre Phase 1b clinical trial in the U.S. and Australia, for CAR T relapsed patients with DLBCL. The study has recently expanded to include and treat CAR T naïve patients diagnosed with a broad range of Non-Hodgkins lymphomas including primary central nervous system lymphoma (PCNSL), chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL), marginal zone lymphoma (MZL), Waldenstrom macroglobulinemia (WM) and follicular lymphoma (FL). Treatment with azer-cel, lymphodepletion (LD) and IL-2 is showing promising results with evidence of meaningful clinical activity, and durability of response. Additionally, the safety profile is manageable and generally well tolerated.

About diffuse large B cell lymphoma (DLBCL)

DLBCL is an aggressive and fast-growing type of non-Hodgkin’s lymphoma (NHL), a type of blood cancer. DLBCL is the most common type of NHL, with approximately 160,000¹ global cases per year and approximately 30,000 new cases per year in the U.S. Relapsed/refractory DLBCL has a high unmet medical need; ~60% of patients treated with approved autologous CD19 CAR T relapse.

¹Science Direct Volume 60, Issue 5, November 2023

About primary central nervous system lymphoma (PCNSL)

PCNSL is a rare and aggressive form of non-Hodgkin lymphoma (NHL), a type of blood cancer that originates in the brain, spinal cord, leptomeninges, or eyes, usually without evidence of systemic disease. In the U.S., there are approximately 1,500 to 1,800 new cases per year with limited approved treatment options and is a high unmet need. Currently, there are no CAR T-cell products approved for the treatment of PCNSL providing a unique opportunity for azer-cel to treat CART naïve patients.

About other types of B Cell Lymphoma

Other subtypes of non-Hodgkin lymphoma (NHL) include chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), the most common slow growing leukemia that can become resistant to therapy; marginal zone lymphoma (MZL), a slow-growing B-cell lymphoma that arises in lymphoid tissues associated with mucosal sites like the stomach and lung; Waldenström macroglobulinemia (WM), a rare slow-growing lymphoma characterized by excess IgM production, which can cause multiple complications ; and follicular lymphoma (FL), a common slow-growing NHL that can become more aggressive. While several targeted therapies and monoclonal antibodies are available for these types of B Cell Lymphoma, relapsed or refractory disease remains an ongoing challenge, highlighting the ongoing need for continued innovation and new and better treatments.

About Interleukin 2 (IL-2)

IL-2 is a cytokine (a protein that affects what happens between cells in the immune system) that helps T-cells (which are part of the immune system that help fight cancer) grow and survive. IL-2 has been shown to help T cells live longer and to enhance the cancer killing functions of CAR T cells, making them more effective at targeting and killing cancer cells.

On September 17, 2025 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported that an abstract was accepted for an oral presentation at the 2025 American Society for Radiation Oncology (ASTRO) Annual Meeting, taking place from September 27 to October 1, 2025, in San Francisco, CA (Press release, Candel Therapeutics, SEP 17, 2025, View Source [SID1234656037]). The oral presentation will feature data from the Company’s phase 3 clinical trial of CAN-2409 in patients with intermediate-to-high-risk localized prostate cancer.

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Details are as follows:

CAN-2409 – Localized Prostate Cancer

Abstract Title: Phase 3, Randomized, Placebo Controlled Clinical Trial of CAN-2409+Prodrug in Combination with Standard of Care External Beam Radiation (EBRT) for Newly Diagnosed Localized Prostate Cancer
Presenter: Glen Gejerman, MD, MBA, Hackensack University Medical Center, Hackensack, NJ
Session Title: SS 03 – GU 1: Advances in Localized Prostate Cancer
Session Date/Time: Sunday, September 28, 2025; 2:30 PM – 2:40 PM PT
Location: Room 24, Moscone Center, San Francisco, CA

The abstract has also been selected for inclusion in ASTRO’s Science Highlights – Genitourinary Cancer session on Sunday, September 28 at 8:00 a.m. PT in Room 24, which will provide a high-level overview of top-rated research in the field.

On September 17, 2025 Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism, reported financial results and provided a business update for the fourth quarter and full fiscal year ended June 30, 2025 (Press release, Rezolute, SEP 17, 2025, View Source [SID1234656036]).

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"We have made substantial progress this year across our two indications for ersodetug in both congenital and tumor hyperinsulinism," said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. "We believe that FDA alignment on a streamlined Phase 3 trial in tumor hyperinsulinism is further recognition of ersodetug’s broad applicability across multiple forms of hyperinsulinism and highlights both the urgent need and the transformative potential of our therapy for patients and families living with this condition. We remain on track to report topline results from the sunRIZE trial in December and look forward to progressing towards potential commercialization."

Recent Pipeline Progress and Anticipated Milestones

Congenital Hyperinsulinism (HI)

· Completed enrollment in sunRIZE, a Phase 3, multicenter, double-blind, randomized, controlled safety and efficacy registrational study of ersodetug for the treatment of congenital HI.

○ Exceeded enrollment with 62 participants enrolled, including approximately 15 percent from U.S. sites.

○ Topline results expected in December 2025.

· Presented "Preliminary Patient Demographics And Baseline Characteristics From A Phase 3 Study (sunRIZE) Of Ersodetug For Hypoglycemia Due To Congenital Hyperinsulinism: Trial In Progress" at the Annual Meeting of the Endocrine Society (ENDO 2025). The enrolled population is comparable to the Phase 2 RIZE study and include:

○ 3.4y average age: 35% <2 years old

○ 15 (average) hypoglycemia events/week

○ 19% daily percent time in hypoglycemia

○ 95% taking ≥1 SOC treatments

Tumor HI

· In August 2025, the Company achieved alignment with FDA on a significantly streamlined clinical development path for its ongoing Phase 3 study (upLIFT) of ersodetug for the treatment tumor HI.

o The truncated study will include as few as 16 participants and will be limited to the single-arm open-label portion of the upLIFT study, removing the need to conduct a double-blind randomized placebo-controlled trial.

o Enrollment is underway and topline results are expected in the second half of 2026.

Corporate Updates

· In August 2025 the Company appointed Dr. Sunil Karnawat as Chief Commercial Officer.

o Dr. Karnawat has over 25 years of experience in global commercialization of biopharmaceuticals and medical devices and will spearhead launch strategy and global market readiness for ersodetug.

o Before joining Rezolute, Dr. Karnawat served as a Vice President at Cytokinetics and Ultragenyx. At Ultragenyx, he was responsible for leading key commercial functions in launching four ultra-rare disease products, including Crysvita.

Fourth Quarter and Full Year Fiscal 2025 Financial Results

Cash, cash equivalents and investments in marketable securities were $167.9 million as of June 30, 2025, compared with $127.1 million as of June 30, 2024.

Research and development (R&D) expenses were $20.9 million for the fourth quarter of fiscal 2025, compared with $19.1 million for the same period a year ago. Full fiscal year 2025 R&D expenses were $61.5 million, compared to $55.7 million in fiscal year 2024. The increase from fiscal year 2024 to fiscal year 2025 was primarily due to (i) increased expenditures in clinical trial activities, (ii) manufacturing costs for ersodetug, and (iii) higher employee-related expenses, which included employee compensation and stock-based compensation.

General and administrative (G&A) expenses were $5.0 million for the fourth quarter of fiscal 2025, compared with $4.0 million for the same period a year ago. Full fiscal year 2025 G&A expenses were $18.4 million, compared to $14.7 million in fiscal year 2024. The increase was primarily attributable to professional fees and employee-related expenses due to increased headcount.

Net loss was $24.4 million for the fourth quarter of fiscal 2025 compared with a net loss of $23.0 million for the same period a year ago. Full year fiscal 2025 net loss was $74.4 million compared to net loss of $68.5 million for the fiscal year 2024.

About Ersodetug

Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor to decrease receptor over-activation by insulin and related substances (such as IGF-2) in the setting of hyperinsulinism (HI), thereby improving hypoglycemia. Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any congenital or acquired form of HI.